Trials Sponsors Conditions Drugs Pricing

Conditions

Hemophilia A

Keywords

Hemophilia A; gene therapy; Adeno-Associated Virus

Outcome Measures

Primary Outcomes (2)

Incidence of dose limiting toxicity (DLT) events
To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) within 10 weeks after administration
10 weeks
The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
To assess the safety of BBM 002 Injection by TEAEs and SAEs
52weeks

Study Arms (1)

Arm of BBM 002

EXPERIMENTAL

1×10\^13 vg/kg, Single-dose treatment.

Genetic: Single dose intravenous injection of BBM 002

Interventions

Single dose intravenous injection of BBM 002Genetic

Single dose intravenous infusion of BBM 002, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene in liver. The dose of BBM 002 will be 1×10\^13 vg/ kg

Arm of BBM 002

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age18 Years+

Sexmale

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Subjects are fully aware of the purpose, nature, methods and possible adverse reactions of the trial and voluntarily sign informed consent.
Males ≥ 18 years of age.

Sponsors & Collaborators

Institute of Hematology & Blood Diseases Hospital, China
Otherlead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

Lei Zhang, MDCONTACT0086-22-23909095 zhanglei1@ihcams.ac.cn

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

Lei Zhang, MD
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
STUDY CHAIR

Central Study Contacts

Lei Zhang, MD

CONTACT

0086-22-23909095 zhanglei1@ihcams.ac.cn

Study Design

Study Type: interventional
Phase: early phase 1
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

July 7, 2022

First Posted

July 12, 2022

Study Start

July 19, 2022

Actual (reported)

Primary Completion

December 15, 2024

Actual (reported)

A Study of FVIII Gene Therapy for Hemophilia A

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Study Arms (1)

Arm of BBM 002

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (2)

Study Arms (1)

Arm of BBM 002

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations